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National MS Society-Supported Researchers Report Early Lab Results of New Approach to Repairing Myelin Damaged by MS

September 30, 2015

  • New York University scientists have uncovered a new approach to stimulating the body’s own resident stem cells to repair nerve-insulating myelin, which is damaged by MS.
  • The researchers, funded in part by the National MS Society, found that by blocking a molecule present in specific stem cells, they were able to stimulate myelin repair and some recovery in mice with an MS-like disease.
  • In the process, they uncovered a new pathway for stimulating myelin repair and are now working to refine the approach to develop a therapy that may be used to repair myelin in people with MS in the future. 
  • The study was published online on September 30, 2015 in the journal Nature.

Background: In MS, the myelin insulation that speeds transmission of signals by nerve fibers is damaged, and the underlying nerve fibers are also destroyed. Some natural repair occurs in people with MS, but at some point this process fails to keep up with the damage. One strategy for restoring nerve function and protecting nerve fibers from destruction is to find a way to stimulate this natural myelin repair process, which is conducted by the body’s own replacement cells, or stem cells, that reside in reservoirs in the adult brain.

This Study:  With funding from the National MS Society and others, Drs. James Salzer, Jayshree Samanta (a former Society postdoctoral fellow) and colleagues at New York University School of Medicine set out to uncover ways to stimulate the body’s own replacement cells, or stem cells, to repair myelin damaged by MS. In a series of studies, the investigators found an unexpected subtype of stem cells in the brain that become activated and transform into myelin-making oligodendrocytes in response to myelin damage. They also found that blocking or eliminating a molecule, called Gli1, which controls the genetic program of these stem cells, they stimulated the cells to repair damaged myelin more effectively.

When they chemically blocked Gli1 in mice with the MS-like disease EAE, they were able to stimulate these stem cells to repair myelin, and the mice experienced some recovery of function.

In the process, the researchers uncovered a new pathway for stimulating myelin repair. 

“These findings identify a new potential avenue for repairing myelin and restoring function in people with MS by mobilizing stem cells within the brain for repair,” said Dr. Salzer. “We are hopeful that further research and development may lead to new therapies based on these findings.”

The study, “Inhibition of Gli1 mobilizes endogenous neural stem cells for remyelination,” was published online on September 30, 2015 in the high-impact journal Nature.

Next Steps:  The molecule the team used to block Gli1, called GANT61, would not likely be potent or specific enough to stimulate repair in people with MS. However, the investigators are now working to develop additional therapy-grade molecules that may more specifically and more potently block Gli1, a necessary step toward developing this lead into a new therapeutic approach to restoring function in people with MS. The National MS Society through Fast Forward plans to support Dr. Salzer’s team to continue development of an enhanced Gli1 blocker for myelin repair.

Read more about research to repair the nervous system

About Multiple Sclerosis

Multiple sclerosis is an unpredictable, often disabling disease of the central nervous system. Symptoms range from numbness and tingling to blindness and paralysis, and there is currently no cure for MS. The progress, severity and specific symptoms of MS in any one person cannot yet be predicted, but advances in research and treatment are leading to better understanding and moving us closer to a world free of MS. An estimated 1 million people live with MS in the United States. Most people with MS are diagnosed between the ages of 20 and 50, and it affects women three times more than men.


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